NCT05230459 A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
| NCT ID | NCT05230459 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | AskBio Inc |
| Condition | Limb Girdle Muscular Dystrophy |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2023-05-15 |
| Primary Completion | 2028-12 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 10 participants in total. It began in 2023-05-15 with a primary completion date of 2028-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
Eligibility Criteria
Inclusion Criteria: 1. Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation. 2. Ability to walk/run 10 meters in \<30 seconds. 3. Able to understand and comply with all study procedures. 4. Sexually active females of childbearing potential and female and male partners of male subjects receiving study intervention must use a barrier method of contraception for the first 6 months after dosing. Exclusion Criteria: 1. Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction \<40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF \>480m/sec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis. 2. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine. 3. Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images. 4. History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and/or abnormal total/direct bilirubin \>upper limit of normal \[ULN\] and/or elevated AST and ALT \>2 ULN). 5. Abnormal renal function (GFR \<60 ml/min, using the Modification of Diet in Renal Disease equation). 6. Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer). 7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits. 8. Requirement for daytime ventilatory support. 9. Change in glucocorticosteroid treatment within 3 months prior to screening visit. 10. Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy. 11. Ongoing participation in any other therapeutic clinical trial. 12. Neutralizing antibody titer to AAV9 \>1:5. 13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05230459 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 65 Years, studying Limb Girdle Muscular Dystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05230459 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05230459 currently recruiting?
Yes, NCT05230459 is actively recruiting participants. Contact the research team at AskFirst@askbio.com for enrollment information.
Where is the NCT05230459 trial being conducted?
This trial is being conducted at Irvine, United States, Iowa City, United States, Kansas City, United States, Baltimore, United States and 2 additional locations.
Who is sponsoring the NCT05230459 clinical trial?
NCT05230459 is sponsored by AskBio Inc. The trial plans to enroll 10 participants.